360 Stem Cell & Regenerative Medicine Newsletter- March 26, 2015

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360 Stem Cell & Regenerative Medicine Newsletter - March 26, 2015

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STEM CELLS Translational Medicine supported this interesting “free range” session at the World Stem Cell Summit that details the progress and future aspirations for translating stem cell therapies to the clinic. The participants include Anthony Atala, MD, Wake Forest University Medical Center; Rick Blume, Excel Venture Management; Geoff Crouse, MBA, MPH, Cord Blood Registry and C. Randal Mills, PhD, California Institute for Regenerative Medicine (CIRM).

Having adequate translation requires the development of infrastructure, resources, and technology to efficiently run clinical trials, evaluate safety and efficacy for new therapies, and develop a process to ensure rapid regulatory approval for new therapies with regulatory agencies. The panel offers insights on the critical role of venture capital, CIRM’s accelerated approach to assist companies and a keen perspective of a top CEO on funding environments.
Semma Therapeutics Announces $44 Million in Funding Led By MPM Capital to Develop Cell Therapy for Treating Type 1 Diabetes: Signs Agreement with Global Pharmaceutical Company Novartis
“Semma Therapeutics, a company developing a cell therapy for Type 1 diabetes, announced that it has closed a $44 million Series A consisting of equity financing and strategic funding. The Series A financing is led by MPM Capital, with Fidelity Biosciences, ARCH Venture Partners, and Medtronic participating. Alongside the equity financing, Semma entered into an undisclosed agreement with Novartis Pharmaceuticals. Doug Melton, PhD, is the Scientific Founder of Semma Therapeutics. He is the Co-Chair of the Department of Stem Cell and Regenerative Biology at Harvard University and is a member of the National Academy of Sciences.”

360 Editor’s note: Dr. Melton is a founding member of the Genetics Policy Institute (GPI) Science Advisory Board. We wish him well in his quest to find effective treatments for diabetes.
University of British Columbia: Stem Cells Show Promise for Reversing Type 2 Diabetes
“Scientists at the University of British Columbia and BetaLogics, part of Janssen Research & Development, LLC have shown for the first time that Type 2 diabetes can be effectively treated with a combination of specially-cultured stem cells and conventional diabetes drugs. The researchers simulated Type 2 diabetes in mice by putting them on a high-fat, high-calorie diet for several weeks. The team then surgically implanted pancreatic-like cells that had been grown in the laboratory from human stem cells. Mice that received a combination of the cells with one of three diabetes drugs became as ‘glucose tolerant’ as the healthy mice, meaning they were able to keep their blood sugar in check, even after ingesting a sugary meal. In contrast, a group of mice with simulated Type 2 diabetes that received the drugs but not the transplants remained glucose-intolerant.” The research was published in Stem Cell Reports.
City of Hope Launches Stem Cell Therapy Clinic to Fight Incurable Disease in New Ways
“City of Hope opened the Alpha Clinic for Cell Therapy and Innovation, patients battling cancer and other life-threatening diseases have another option: stem cell-based therapy. The Alpha Clinic is funded by an $8 million grant from the California Institute for Regenerative Medicine (CIRM) that will be supplemented by matching funds from City of Hope. It will combine the uniquely patient-centered care for which City of Hope is known with the most innovative, stem cell-based therapies being studied to date. The approach is expected to revolutionize the treatment of not only cancer, but also AIDS and other life-threatening diseases.”
World Stem Cell Summit
Life Sciences Report: Edward Lanphier - Why the Future of Regenerative Medicine is Worth Fighting For
“Stem cell, gene therapy and genetically modified cell therapy companies have begun to attract serious capital from investors who understand biotechnology and have the wherewithal to move products into the clinic and into the market. But battles still loom on the political and reimbursement front. Edward Lanphier, founder and CEO of Sangamo BioSciences Inc. and current chairman of the Alliance for Regenerative Medicine, sees near-term milestones on the horizon, as well as additional inflows of validating capital from prominent investors.” In this interview with The Life Sciences Report, industry thought- leader Lanphier describes upcoming milestones and share-moving catalysts.
International Society for Stem Cell Research (ISSCR) Issues Statement on Human Germline Genome Modification
“The ISSCR calls for a moratorium on attempts to apply nuclear genome editing of the human germ line in clinical practice. Scientists currently lack an adequate understanding of the safety and potential long -term risks of germline genome modification. Moreover, the ISSCR asserts that a deeper and more rigorous deliberation on the ethical, legal and societal implications of any attempts at modifying the human germ line is essential if its clinical practice is ever to be sanctioned.”
Wake Forest
Mount Sinai Researchers Discover Genetic Origins of Myelodysplastic Syndrome Using Stem Cells
“Induced pluripotent stem cells (iPSCs)—adult cells reprogrammed back to an embryonic stem cell-like state—may better model the genetic contributions to each patient's particular disease. In a process called cellular reprogramming, researchers at Icahn School of Medicine at Mount Sinai have taken mature blood cells from patients with myelodysplastic syndrome (MDS) and reprogrammed them back into iPSCs to study the genetic origins of this rare blood cancer. The results appear in an upcoming issue of Nature Biotechnology.”
Mayo Clinic
Scripps Discovers Enzyme that Keeps Blood Stem Cells Functional to Prevent Anemia
Researchers at The Scripps Research Institute (TSRI) discovered that a particular enzyme in hematopoietic stem cells (HSCs) is key to maintaining healthy periods of inactivity. Their findings show that animal models without this enzyme experience dangerous HSC activation and ultimately succumb to lethal anemia. With this new understanding of the enzyme scientists are closer to improving therapies for diseases such as bone marrow failure syndrome, anemia, leukemia, lymphoma and immunodeficiencies. The research was published in the journal Blood.
Neuralstem Announces Topline Results Of Phase II ALS Trial
Neuralstem, Inc. reported top line data from the Phase II trial of NSI-566 spinal cord-derived neural stem cells under development for the treatment of amyotrophic lateral sclerosis (ALS). According to the company announcement, the study met primary safety endpoints. The open-label, dose-escalating trial treated 15 ambulatory patients, divided into 5 dosing cohorts, at three centers: Emory University Hospital in Atlanta, Georgia, the ALS Clinic at the University of Michigan Health System and Massachusetts General Hospital. The clinical trial is directed by principal investigator, Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System.

“The collaborative research program between L’Oréal and the Centre for Inflammation and Tissue Homeostasis (CITH) at inStem will focus on exploration of biological mechanisms that influence skin-aging. A major objective of this collaboration would be to study the molecular mechanisms and signaling pathways involved in the cell biology of skin homeostasis. L’Oréal will share its active ingredients for evaluation and validation of these concepts.”
California’s Stem Cell Agency Adds Online Advocacy Toolbox and Revises Website
Kevin McCormack, Sr. Director Public Communications & Patient Advocate Outreach of the California Institute for Regenerative Medicine informed the 360 Newsletter that CIRM has added a very useful advocacy toolbox to its updated its user friendly website. Check it all out! Here's a link to the Toolbox: https://www.cirm.ca.gov/patients/patients-toolbox Here's a link to a blog describing the Toolbox: http://blog.cirm.ca.gov/2015/03/24/the-best-tools-to-be-the-best-advocate/
New StemBioSys IP a Key Step in Moving to Product Commercialization
In a company media release, StemBioSys Inc. announced that has received patent approval (US Patent #8,961,955) that expands coverage of technology licensed from the University of Texas Health Science Center at San Antonio.

"The patent entitled ‘Maintenance and Propagation of Mesenchymal Stem Cells’ is an extension of the company’s core technology which is directed at enhancing the isolation, expansion and delivery of various types of stem cells. The new patent covers application of the company technology for bone healing applications, an area which StemBioSys is actively pursuing as one of many potential therapeutic applications for the technology."

360 Newsletter Editor

Bernard Siegel
Executive Director
Genetics Policy Institute

Stem Cells and the Transformative Power of Hope-a TEDx talk by Bernard Siegel, Editor of 360 Newsletter
Domain: Medical
Category: Biology

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